The table below is a review of notable updates that occurred in June 2022 for investigational products in development (not an inclusive list). Click on the status link to view our full coverage.
| Drug | Pharmacologic Class | Proposed Indication | Status |
| Cardiovascular Disease | |||
| Landiolol (Eagle Pharmaceuticals, Inc.) | Ultra-short acting, cardioselective, beta-1 adrenoreceptor blocker | Short-term reduction of ventricular rate in patients with supraventricular tachycardia, including atrial fibrillation and atrial flutter. | NDA submitted |
| Omecamtiv mecarbil (Cytokinetics, Inc.) | Selective small molecule cardiac myosin activator | Treatment of heart failure with reduced ejection fraction. | PDUFA target date has been extended |
| Hematological Disorders | |||
| Betibeglogene autotemcel (beti-cel; bluebird bio, Inc.) | One-time gene therapy that adds functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. | Treatment of patients with beta-thalassemia who require regular red blood cell transfusion. | FDA panel voted in favor of approval |
| Elivaldogene autotemcel (eli-cel; bluebird bio, Inc.) | One-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells. | Treatment of early active cerebral adrenoleukodystrophy in patients less than 18 years of age who do not have an available and willing HLA-matched sibling HSC donor. | FDA panel voted in favor of approval |
| VLX-1005 (Veralox Therapeutics) | Selective small molecule 12-LOX inhibitor | Treatment and prevention of heparin-induced thrombocytopenia and thrombosis. | Fast Track designation |
| Immunization | |||
| NVX-CoV2373 (Novavax) | Protein-based COVID-19 vaccine | Active immunization for the prevention of COVID-19 for individuals 18 years of age and older. | FDA panel voted in favor of granting EUA |
| Infectious Diseases | |||
| Nirmatrelvir + ritonavir (Paxlovid; Pfizer Inc.) | SARS-CoV-2 main protease inhibitor + HIV-1 protease inhibitor | Treatment of COVID-19 in both vaccinated and unvaccinated individuals who are at high risk for progression to severe illness from COVID-19. | NDA submitted |
| Tebipenem HBr (tebipenem pivoxil hydrobromide; Spero Therapeutics) | Oral antibiotic in carbapenem class | Treatment of adults with complicated urinary tract infections, including pyelonephritis, caused by susceptible microorganisms. | Complete Response Letter issued |
| Musculoskeletal Disorders | |||
| BCX9250 (BioCryst Pharmaceuticals, Inc.) | Activin receptor-like kinase-2 (ALK-2) inhibitor | Prevention of heterotopic ossification in patients with fibrodysplasia ossificans progressiva. | Fast Track designation |
| Neurologic Disorders | |||
| AMX0035 (sodium phenylbutyrate and taurursodiol; Amylyx Pharmaceuticals, Inc.) | Oral, fixed-dose coformulation targeting endoplasmic and mitochondrial-dependent neuronal degeneration pathways | Treatment of amyotrophic lateral sclerosis (ALS). | PDUFA target date has been extended |
| ATX01 (topical amitriptyline; AlgoTx) | Directly inhibits pain signaling in the nerve fibers on the epidermis and dermis | Treatment of chemotherapy-induced neuropathic pain. | Fast Track designation |
| Pimavanserin (Acadia Pharmaceuticals Inc.) | Atypical antipsychotic | Treatment of hallucinations and delusions associated with Alzheimer disease psychosis. | FDA panel voted against approval |
| Ublituximab (TG Therapeutics, Inc.) | Glycoengineered monoclonal antibody targeting CD20 | Treatment of patients with relapsing forms of multiple sclerosis. | PDUFA target date has been extended |
| Ob/Gyn | |||
| Myfembree (relugolix, estradiol, and norethindrone acetate; Myovant Sciences) | GnRH antagonist + estrogen + progestin | To include long term data in premenopausal women with heavy menstrual bleeding associated with uterine fibroids. | sNDA accepted for review |
| Oncology | |||
| Omburtamab (Y-mAbs Therapeutics, Inc.) | Radiolabeled monoclonal antibody that targets tumor cells expressing B7-H3 | Treatment of pediatric patients with CNS/leptomeningeal metastasis form neuroblastoma | BLA accepted for Priority Review |
| VBI-1901 (VBI Vaccines Inc.) | Bivalent eVLP vaccine immunotherapeutic candidate that targets 2 highly immunogenic CMV antigens, gB and pp65. | Treatment of glioblastoma. | Orphan Drug designation |
| Ophthalmic Disorders | |||
| Aflibercept (Eylea Regeneron Pharmaceuticals, Inc.) | Recombinant fusion protein (human VEGF inhibitor + human IgG1) | Treatment of diabetic retinopathy with every 16-week dosing regimen. | sBLA accepted for review |
| Respiratory Disorders | |||
| AD109 (atomoxetine + aroxybutynin; Apnimed) | SNRI + selective antimuscarinic | Treatment of obstructive sleep apnea. | Fast Track designation |
